With RTA 801, Reata has proven its ability to discover small molecule chaperones that stabilize proper folding

RTA 801 - SOD1 Protein Folding Stabilizer

Superoxide dismutase 1 (SOD1) is a well-accepted target for the devastating neurodegenerative disease amyotrophic lateral sclerosis (ALS), commonly known as Lou Gehrig’s disease. Reata has been using its innovative protein folding drug discovery platform to identify novel molecules that can act as molecular chaperones to stabilize misfoled SOD1 and therefore affect the ALS disease process.

RTA 801 is the first SOD1 folding stabilizer to emerge from this discovery program. It was selected for preclinical development based on superior potency in in vitro[r4] assays, and has demonstrated promising activity in initial animal studies.

ALS Development Program
Preclinical testing of RTA 801 has demonstrated a promising profile of activity.
	RTA 801 showed potent in vitro activity in Reata's drug discovery assay and in subsequent secondary assays. It was shown biochemically to stabilize SOD1, likely through direct binding.
	Pharmacokinetic and tissue analysis of RTA 801 indicates that the drug is orally bioavailable and crosses the blood-brain barrier.
	RTA 801 was well tolerated in multi-dose rodent toxicology studies.

Should further animal studies continue to indicate that this drug has promising activity, RTA 801 will be moved into clinical development.